估算美国使用多奈单抗对早期症状性阿尔茨海默病进行限期治疗的经济合理价格。

IF 3.9 3区医学 Q1 CLINICAL NEUROLOGY Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-09-18 DOI:10.1007/s40120-024-00649-y

Malaz Boustani, Erin G Doty, Louis P Garrison, Lee J Smolen, Timothy M Klein, Daniel R Murphy, Andrew W Spargo, Mark Belger, Joseph A Johnston

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引用次数: 0

摘要

简介：本经济模型的目的是根据 TRAILBLAZER-ALZ 2 3 期试验（NCT04437511）的临床数据，估算在美国使用多那尼单抗治疗早期症状性阿尔茨海默病（AD）的经济合理价格（EJP）：我们改编了临床与经济研究所（Institute for Clinical and Economic Review）开发的AD马尔可夫状态转换模型，从医疗保健系统的角度和作为共同基础案例的社会角度估算了多那尼单抗在不同支付意愿（WTP）阈值下的EJP：假设每个质量调整生命年（QALY）获得的WTP阈值为150,000美元，那么从医疗保健系统的角度来看，模型估计多那尼单抗1年（13剂）的EJP为80,538美元，从社会角度来看为91,126美元；如果每个质量调整生命年获得的WTP阈值为100,000美元，那么从医疗保健系统的角度来看，模型估计多那尼单抗1年（13剂）的EJP为44,691美元，从社会角度来看为55,419美元。根据联合基础病例分析中患者接受多那尼单抗的平均剂量，从医疗保健系统角度得出的每 QALY 收益 EJP 为 150,000 美元时，每位患者的平均治疗总成本估计为 77,812 美元。单向敏感性分析（OWSA）表明，治疗效果、开始治疗时的疾病严重程度和治疗效果持续时间是潜在 EJP 的主要驱动因素：以 TRAILBLAZER-ALZ 2 研究为基础的模型模拟结果表明，使用多那尼单抗进行限期治疗的 EJP 超过了目前可用的淀粉样蛋白靶向疗法的每剂量清单价格，这意味着可能会降低终生成本并提高性价比。

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Estimating the Economically Justifiable Price of Limited-Duration Treatment with Donanemab for Early Symptomatic Alzheimer's Disease in the United States.

Introduction: The goal of this economic model is to estimate an economically justifiable price (EJP) for using donanemab for the treatment of early symptomatic Alzheimer's disease (AD) in the United States based on clinical data from the phase 3 TRAILBLAZER-ALZ 2 trial (NCT04437511).

Methods: We adapted an AD Markov state-transition model developed by the Institute for Clinical and Economic Review to estimate the EJP for donanemab at different willingness-to-pay (WTP) thresholds from the health care system perspective and the societal perspective as co-base cases.

Results: Assuming a WTP threshold of $150,000 per quality-adjusted life-year (QALY) gained, the model estimates a 1-year (13-dose) EJP for donanemab of $80,538 from the health care system perspective and $91,126 from the societal perspective; at a WTP threshold of $100,000 per QALY gained, the model estimates a 1-year (13-dose) EJP for donanemab of $44,691 from the health care system perspective and $55,419 from the societal perspective. Mean total treatment costs per patient at the $150,000 per QALY gained EJP derived from the health care system perspective were estimated at $77,812 based on the average number of doses of donanemab patients received in the co-base case analysis. One-way sensitivity analysis (OWSA) indicated that treatment efficacy, disease severity at the time of treatment initiation, and duration of treatment effect were the main drivers of the potential EJP.

Conclusions: Results from this modeling simulation informed by the TRAILBLAZER-ALZ 2 study support an EJP for limited-duration treatment with donanemab that exceeds per-dose list prices for currently available amyloid-targeting therapies, implying potentially lower lifetime costs and better value for money.

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来源期刊

Neurology and Therapy CLINICAL NEUROLOGY-

CiteScore

5.40

自引率

8.10%

发文量

103

审稿时长

6 weeks

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